News

Proteostasis Therapeutics has filed an Investigational New Drug application with the U.S. Food and Drug Administration for PTI-808 as a treatment for cystic fibrosis. PTI-808 promotes the activity of the cystic fibrosis transmembrane conductance regulator, or CFTR, the protein that is faulty in CF. Scientists call the drug a potentiator…

Cystic Fibrosis Foundation Therapeutics (CFFT) will increase funding to support Spyryx Biosciences‘ Phase 2 trial assessing SPX-101 in people with cystic fibrosis. The funding is part of an award Spyryx received in 2015 from CFFT, the Cystic Fibrosis Foundation‘s…

Children with cystic fibrosis (CF) have less sleep and more sleep interruptions than healthy children, even when the disease is well-managed and stable, an Australian study indicates. The research, “How Well Do Children with Cystic Fibrosis Sleep? An Actigraphic and Questionnaire-Based Study,” was published in The Journal of…

Forty cystic fibrosis patients or family members are winners of Vertex Pharmaceuticals‘ first All in for CF scholarships. The awards are for $5,000, making the total for the 2017-2018 academic year $200,000. Boston-based Vertex is a biotech company that develops therapies for cystic fibrosis and  other life-threatening conditions. The company…

So-called silent gene mutations in the cystic fibrosis (CF)-causing CFTR gene are not necessarily silent at all. A research team discovered that one such mutation also causes the resulting protein to have a poor function. The insights put the spotlight on the complexity of CF genetic causes, but also advances…

Abiding by the extensive treatment regimen for cystic fibrosis (CF) can be especially challenging for pediatric patients. A new study found that frequent monitoring of pulmonary function tests (PFTs) and medication reminders can help pediatric CF patients better meet their daily medication and vitamin…

Celtaxsys announced it has completed enrollment of its Phase 2b clinical trial evaluating the company’s lead anti-inflammatory candidate, oral acebilustat, in adult patients with cystic fibrosis (CF). Acebilustat (formerly known as CTX-4430) is a once-daily, oral anti-inflammatory being evaluated for its safety and efficacy in treating diseases like CF. It is a new small…

X-Chem Pharmaceuticals and Vertex Pharmaceuticals will jointly develop small molecules targeting cystic fibrosis (CF) and other severe genetic diseases. Under the collaboration announced May 16, X-Chem will apply its proprietary DEX libraries — which holds more than 120 billion small compounds — to discover new drug candidates against targets involved in CF…

The U.S. Food and Drug Administration (FDA) has expanded its approval of Kalydeco (ivacaftor) to include cystic fibrosis (CF) patients with one of 23 additional rare mutations — more than tripling the number of mutations the oral drug may be used to treat from 10 to 33. But the decision’s…

Spyryx Biosciences will present two studies in Washington this week on SPX-101‘s ability to treat cystic fibrosis by clearing mucus from airways. One of the preclinical-trial studies it will discuss at the American Thoracic Society‘s annual convention showed that SPX-101 improved survival in several models of CF-like lung diseases.